AR126271A1 - LEUCINE-RICH REPEATED KINASE 2 (LRRK2) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF - Google Patents
LEUCINE-RICH REPEATED KINASE 2 (LRRK2) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOFInfo
- Publication number
- AR126271A1 AR126271A1 ARP220101700A ARP220101700A AR126271A1 AR 126271 A1 AR126271 A1 AR 126271A1 AR P220101700 A ARP220101700 A AR P220101700A AR P220101700 A ARP220101700 A AR P220101700A AR 126271 A1 AR126271 A1 AR 126271A1
- Authority
- AR
- Argentina
- Prior art keywords
- lrrk2
- pharmaceutical composition
- dsrna agent
- dsrna
- agent
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 4
- 108091000080 Phosphotransferase Proteins 0.000 title 1
- 102000020233 phosphotransferase Human genes 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 13
- 101000941879 Homo sapiens Leucine-rich repeat serine/threonine-protein kinase 2 Proteins 0.000 abstract 7
- 102100032693 Leucine-rich repeat serine/threonine-protein kinase 2 Human genes 0.000 abstract 7
- 239000008194 pharmaceutical composition Substances 0.000 abstract 7
- 230000002401 inhibitory effect Effects 0.000 abstract 5
- 210000004027 cell Anatomy 0.000 abstract 4
- 101100021877 Homo sapiens LRRK2 gene Proteins 0.000 abstract 3
- 101150081013 LRRK2 gene Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 229920002477 rna polymer Polymers 0.000 abstract 2
- 102000016349 Myosin Light Chains Human genes 0.000 abstract 1
- 108010067385 Myosin Light Chains Proteins 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 208000018737 Parkinson disease Diseases 0.000 abstract 1
- 108091081021 Sense strand Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 150000002632 lipids Chemical class 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
Abstract
La divulgación se refiere a agentes de ácido ribonucleico bicatenario (dsRNAi) y composiciones que se dirigen al gen quinasa 2 repetida rica en leucina (LRRK2), así como a métodos para inhibir la expresión de un gen LRRK2 y métodos para tratar a pacientes que tienen una enfermedad o trastorno asociado a LRRK2, p. ej., la enfermedad de Parkinson, utilizando tales agentes y composiciones de dsRNAi. Reivindicación 1: Un agente de ácido ribonucleico bicatenario (dsRNA) para inhibir la expresión de LRRK2, caracterizado porque el agente de dsRNA comprende una cadena sentido y una cadena antisentido que forman una región bicatenaria, donde la cadena antisentido comprende una región de complementariedad con un mRNA que codifica LRRK2, y donde la región de complementariedad comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de una cualquiera de las secuencias de nucleótidos en antisentido en una cualquiera de las Tablas 3 a 7. Reivindicación 89: Un agente dsRNA caracterizado porque se dirige a una región caliente del mRNA (LRRK2) de una proteína que interactúa con la cadena ligera reguladora de la miosina. Reivindicación 90: Una célula caracterizada porque contiene el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89. Reivindicación 91: Una composición farmacéutica para inhibir la expresión de un gen que codifica LRRK2, caracterizada porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89. Reivindicación 92: Una composición farmacéutica caracterizada porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89 y una formulación lipídica. Reivindicación 98: Un método para inhibir la expresión de un gen LRRK2 en una célula, el método caracterizado porque comprende contactar la célula con el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89, o la composición farmacéutica de una cualquiera de las reivindicaciones 91 a 97, inhibiendo así la expresión del gen LRRK2 en la célula. Reivindicación 120: Un equipo caracterizado porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89 o la composición farmacéutica de una cualquiera de las reivindicaciones 91 a 97. Reivindicación 121: Un vial caracterizado porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89 o la composición farmacéutica de una cualquiera de las reivindicaciones 91 a 97. Reivindicación 122: Una jeringa caracterizada porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89 o la composición farmacéutica de una cualquiera de las reivindicaciones 91 a 97. Reivindicación 123: Una bomba intratecal caracterizada porque comprende el agente de dsRNA de una cualquiera de las reivindicaciones 1 a 89 o la composición farmacéutica de una cualquiera de las reivindicaciones 91 a 97.The disclosure relates to double-stranded ribonucleic acid (dsRNAi) agents and compositions that target the leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods for inhibiting the expression of an LRRK2 gene and methods for treating patients who have a disease or disorder associated with LRRK2, e.g. e.g., Parkinson's disease, using such agents and dsRNAi compositions. Claim 1: A double-stranded ribonucleic acid (dsRNA) agent for inhibiting the expression of LRRK2, characterized in that the dsRNA agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein the antisense strand comprises a region of complementarity with a mRNA encoding LRRK2, and wherein the region of complementarity comprises at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from any one of the antisense nucleotide sequences in any one of Tables 3 to 7. Claim 89: An agent dsRNA characterized because it targets a hot region of the mRNA (LRRK2) of a protein that interacts with the myosin regulatory light chain. Claim 90: A cell characterized in that it contains the dsRNA agent of any one of claims 1 to 89. Claim 91: A pharmaceutical composition for inhibiting the expression of a gene encoding LRRK2, characterized in that it comprises the dsRNA agent of any one of claims 1 to 89. Claim 92: A pharmaceutical composition characterized in that it comprises the dsRNA agent of any one of claims 1 to 89 and a lipid formulation. Claim 98: A method for inhibiting the expression of an LRRK2 gene in a cell, the method characterized in that it comprises contacting the cell with the dsRNA agent of any one of claims 1 to 89, or the pharmaceutical composition of any one of claims 91 to 97, thus inhibiting the expression of the LRRK2 gene in the cell. Claim 120: A kit characterized in that it comprises the dsRNA agent of any one of claims 1 to 89 or the pharmaceutical composition of any one of claims 91 to 97. Claim 121: A vial characterized in that it comprises the dsRNA agent of any one of claims 1 to 89 or the pharmaceutical composition of any one of claims 91 to 97. Claim 122: A syringe characterized in that it comprises the dsRNA agent of any one of claims 1 to 89 or the pharmaceutical composition of any one of the Claims 91 to 97. Claim 123: An intrathecal pump characterized in that it comprises the dsRNA agent of any one of claims 1 to 89 or the pharmaceutical composition of any one of claims 91 to 97.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063353953P | 2020-06-21 | 2020-06-21 | |
| US202163216119P | 2021-06-29 | 2021-06-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR126271A1 true AR126271A1 (en) | 2023-10-04 |
Family
ID=88679262
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220101700A AR126271A1 (en) | 2020-06-21 | 2022-06-29 | LEUCINE-RICH REPEATED KINASE 2 (LRRK2) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR126271A1 (en) |
-
2022
- 2022-06-29 AR ARP220101700A patent/AR126271A1/en unknown
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